Alerta

RNA-LOADED LIPID NANOPARTICLES

Resumen de: AU2024285136A1

Compositions are provided that include a lipid nanoparticle (LNP);a self-replicating RNA encoding a gene of interest loaded within the LNP; and an inhibitory nucleic acid, such as a small interfering RNA (siRNA) targeting IFN-α/β receptor l(Ifharl) gene loaded within the LNP, and use of the compositions for generating an immune response.

METHODS AND COMPOSITIONS FOR MODULATING PLASMINOGEN ACTIVATOR INHIBITOR-1 (PAI-1)

Resumen de: AU2024280598A1

A siRNA molecule for inhibiting expression of plasminogen activator inhibitor-1 (PAI-1) is disclosed and may have a sense strand and an antisense strand. The sense strand may have at least 80% sequence identity to any one of SEQ ID NOs: 3, 5, 7, and 9. In some embodiments, the antisense strand may have at least 80% sequence identity to any one of SEQ ID NOs: 4, 6, 8, and 10. The sense strand and/or the antisense strand may comprise one or more modified nucleotides. The sense strand and/or the antisense strand may comprise of a ligand. A lipid nanoparticle comprising the siRNA molecule is also disclosed. Methods of using the siRNA molecule or the lipid nanoparticle comprising the siRNA molecule to treat a subject who suffers from at least one PAI-1-associated condition, disease or disorder are also disclosed.

GENE EDITING SYSTEMS AND COMPOSITIONS FOR TREATMENT OF HEMOGLOBINOPATHIES AND METHODS OF USING THE SAME

Resumen de: AU2024272060A1

The present disclosure describes improved LNP-based nucleobase editing systems and therapeutics for use in treating hemoglobinopathies, including sickle cell disease and beta-thalassemia. In particular, the disclosure describes improved LNPs, including novel and improved ionizable lipids for making LNPs, that enhance the targeted delivery of LNP-based nucleobase editing systems and therapeutics to red blood cell progenitor cells, enabling treatment of hemoglobinopathies, in vivo.

IONIZABLE AMINE LIPIDS

Resumen de: AU2024275548A1

The disclosure provides ionizable lipids and lipid nanoparticle (LNP) compositions comprising ionizable lipids, helper lipids, neutral lipids, and PEG lipids useful for the delivery of biologically active agents, for example delivering biologically active agents to cells to prepare engineered cells. The LNP compositions disclosed herein are useful in methods of gene editing and methods of delivering a biologically active agent and methods of modifying or cleaving DNA.

METHODS OF PRODUCING EXTRACELLULAR VESICLES, EXTRACELLULAR VESICLES AND USES THEREOF

Resumen de: AU2024272799A1

The present invention provides a method of producing extracellular vesicles (EVs) comprising incubating or culturing EV producing cells in media. The present invention also provides a population of EVs and compositions comprising the vesicles and methods and uses thereof.

EXTRACELLULAR VESICLES

Resumen de: AU2024272694A1

The present disclosure relates to microRNAs (miRs) and extracellular vesicles comprising same and therapeutic uses thereof, for example, in modulating angiogenesis.

ＨＰＶ感染関連疾患を予防又は治療するためのポリヌクレオチド分子

Resumen de: MX2025007651A

The present application relates to a polynucleotide molecule that can be used for preventing or treating HPV infection-related diseases, and a pharmaceutical product, a pharmaceutical composition, or an mRNA vaccine comprising said polynucleotide.

ANELLOSOMES FOR DELIVERING SECRETED THERAPEUTIC MODALITIES

Resumen de: US20260009050A1

This invention relates generally to anellosomes and compositions and uses thereof.

CATIONIC LIPID COMPOUND WITH HIGH EFFICIENCY, LOW TOXICITY AND STABLE EXPRESSION, AND COMPOSITION COMPRISNG SAME

Resumen de: US20260008746A1

The present invention provides a cationic lipid compound with high efficiency, low toxicity and stable expression, which is a compound of formula (I) or an N-oxide, a solvate, a pharmaceutically acceptable salt or a stereoisomer thereof Further provided are a composition comprising the described compound and a use thereof in delivering a therapeutic or prophylactic agent.

LIPIDS CONJUGATED WITH POLY(ETHYLENE OXIDE) HAVING C1 TO C3-ALKYLOXYMETHYL SIDE CHAINS

Resumen de: US20260008887A1

The present invention refers to polyoxyalkylene based compounds and their manufacturing method as well as compositions including at least one polyoxyalkylene based compound and at least one active agent. Furthermore, the present invention refers to the manufacture of the compositions of the present invention as well as their use for the treatment of an illness in mammals or humans.

NANOPARTICLE COMPOSITIONS AND METHODS FOR BIOLOGICAL MEASUREMENTS

Resumen de: WO2026011085A1

This disclosure provides nanoparticle compositions, and use thereof for isolating and measuring proteins, protein degrader action, and cells. The disclosed nanoparticles can be identified by barcodes that reveal the identity of and post translational modifications to the bound protein using image processing techniques described herein.

PLACENTA-TARGETED NANOPARTICLES OR CONJUGATES AND METHODS OF USE THEREOF

Resumen de: WO2026010906A1

Provided herein are compositions, methods, and particles that include a peptide that targets placental chondroitin sulfate A (placental CSA), wherein the amino acid sequence of the polypeptide is as set forth in SEQ ID NO: 1 (EDVKDINFDTKEKFLAGLIVSFHEGKC).

USE OF LIPID NANOPARTICLE MRNA FOR TERT SPECIFIC CANCER IMMUNOTHERAPY

Resumen de: WO2026010842A1

The present invention relates to compositions for inducing an immune response against a tumor antigen in a subject, the composition comprising at least one mRNA encoding at least one tumor antigen selected from the group consisting of hTERT, mTERT, and KRASG12D, encapsulated in a lipid nanoparticle.

PHARMACEUTICAL FORMULATION WITH IMPROVED SKIN PERMEABILITY

Resumen de: WO2026010674A1

The present invention discloses a formulation with improved skin permeability. Particularly, the invention is related to a formulation in the form of nanoemulsion comprising at least one lubricant, saponin, oil component, surfactant and at least one pharmaceutical or nutraceutical active ingredient with improved skin permeability.

IONIZABLE CATIONIC LIPIDS INCORPORATING SILICON

Resumen de: US20260008793A1

The invention relates to a novel ionizable cationic lipid family incorporating silicon, which belongs to the trademark LipexSil™ 1st generation lipids wherein at least one of the two side chains contains silyl acetal linker. Lipids containing silyl acetal linker(s) so far are unprecedented in the art and are effective as ionizable cationic lipids in the formulation of empty or loaded lipid nanoparticles (LNPs). The novel linkers according to the invention are designed by means of borane catalysts WO 2022/129966. The invention describes the synthesis of the lipids of formula (I), formation and characterization of nanoparticles and biological experiments demonstrating that the lipid nanoparticles prepared with these novel lipids can efficiently deliver their cargo (e.g. RNA, DNA, mRNA, siRNA, pDNA, circular DNA, small biologically active molecules) into the cells.

CONJUGATION LINKERS

Resumen de: US20260007768A1

Provided are β-eliminative linkers suitable for the conjugation of small molecule, peptide, and protein and compounds comprising the linkers.

COMPOSITIONS AND METHODS FOR TREATING ALPHA-1 ANTITRYPSIN DEFICIENCY

Resumen de: US20260007772A1

Compositions and methods for expressing alpha 1 antitrypsin (AAT) in a host cell are provided. Also provided are compositions and methods for treating subjects having alpha 1 antitrypsin deficiency (AATD).

THROMBOLYTIC AND NEURO/VASCULOPROTECTIVE NANOCONJUGATES

Resumen de: US20260007614A1

Provided herein are compositions, systems, kits, and methods for treating a patient with a thromboembolism by administering nanoconjugates comprising nanoparticles encapsulating and/or conjugated to: i) tissue-type plasminogen activator (tPA), and ii) at least one antioxidant enzyme selected from the group consisting of: superoxide dismutase, glutathione peroxidase, glutathione reductase, and a catalase.

Novel Ionizable Cationic Lipids

Resumen de: US20260007612A1

Provided herein, inter alia, novel ionizable cationic lipids, lipid nanoparticles comprising the novel ionizable cationic lipids and methods of using said nanoparticles.

USE OF A SPRAY FREEZE-DRYING PROCESS FOR THE LYOPHILIZATION OF A mRNA-ENCAPSULATING LIPID NANOPARTICLES FORMULATION

Resumen de: US20260007604A1

The invention relates to the lyophilization of a liquid pharmaceutical formulation including lipid nanoparticles encapsulating mRNA.According to the invention, a spray freeze-drying process is used to achieve the lyophilization.The invention is of particular interest for the lyophilization of mRNA vaccine formulations.

LONG-ACTING LOW-TOXICITY NOVEL CATIONIC LIPID COMPOUNDS AND COMPOSITION THEREOF

Resumen de: US20260007613A1

Provided in the present invention are long-acting low-toxicity novel cationic lipid compounds, which are compounds shown as formula (I), or N-oxides, solvates, pharmaceutically acceptable salts or stereoisomers thereof. Further provided are a composition comprising the compounds and the use thereof for the delivery of therapeutic or prophylactic agents.

COMPOSITIONS AND METHODS FOR DELIVERY OF RNA

Resumen de: US20260007769A1

The disclosure provides nanoemulsion compositions and methods of making and using thereof to deliver a bioactive agent such as a nucleic acid to a subject. The nanoemulsion composition comprises a hydrophobic core based on inorganic nanoparticles in a lipid nanoparticle that allows imaging as well as delivering nucleic acids. Methods of using these particles for treatment and vaccination are also provided.

MULTI-LAYER NANOPARTICLES FOR NUCLEIC ACID DELIVERY

Resumen de: WO2026010484A1

The present invention relates to multi-layer nanoparticles for nucleic acid delivery, a method for preparing same, a nucleic acid delivery composition comprising same, an antibacterial composition, and a pharmaceutical composition for preventing or treating infectious diseases. The multi-layer nanoparticles for nucleic acid delivery comprise: gold nanoparticles; a polyethyleneimine modified on the surface of the gold nanoparticles; and chitosan applied on the gold nanoparticles modified with the polyethyleneimine. According to the present invention, the introduction of nucleic acids (e.g., ASOs) into target bacterial cells is maximized, and highly efficiently loaded nucleic acids can be effectively delivered into bacteria to maximize the effect of inhibiting target nucleic acids, and thus the present invention can be useful as an antibacterial agent and a gene therapeutic agent.

DUAL TARGET FERRITIN-PROTEIN COMPLEX AND USE THEREOF

Resumen de: WO2026010421A1

The present invention relates to a ferritin-protein complex capable of dual targeting and a use thereof. The dual-target ferritin-protein complex of the present invention can selectively deliver a drug only to tumor tissues and cancer cells, thereby increasing the efficacy of the drug.

PHARMACEUTICAL LIPID NANOPARTICLE COMPOSITION WITH IMPROVED TARGETED DELIVERY ABILITY AND SAFETY

Nº publicación: WO2026010380A1 08/01/2026

Solicitante:

RESEARCH \uFF06 BUSINESS FOUNDATION SUNGKYUNKWAN UNIV [KR]
KOREA INSTITUTE OF SCIENCE AND TECH [KR]
MVRIX CO LTD [KR]
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